Anemia is the most frequent peripheral
cytopenia observed in
myelodysplastic syndromes (MDS) and has been recognized among the most important factors affecting the outcome of patients with MDS. In patients who are not candidates for potentially curative approaches, therapeutic options for symptomatic
anemia include red blood cell (RBC) transfusion and
iron chelation, hematopoietic
growth factors, immunosuppression, immune-modulatory drugs, and hypomethylating agents. In about 40% of patients, regular RBC transfusions are the only therapeutic option that can be offered. The onset of a regular transfusion requirement significantly worsens the survival of patients with MDS. Transfusion-dependent patients invariably develop secondary
iron overload.
Elevated serum ferritin was proven to be associated with worse survival in transfusion-dependent patients, and recent data obtained using magnetic resonance imaging show both hepatic and myocardial
iron accumulation in heavily transfused patients. According to evidence-based guidelines, patients with
sideroblastic anemia,
5q- syndrome, or other forms of
refractory anemia, in whom long-term transfusion
therapy is likely, are recognized as the best candidates to receive
iron chelation therapy. In addition, patients who are candidates for allogeneic
stem cell transplantation might also benefit from
chelation therapy because
iron overload is associated with increased
transplantation-related mortality. RBC transfusions and
iron chelation are the mainstay of
therapy for many individuals with MDS. However, critical issues remain to be clarified in order to optimize treatment, including the identification of target
hemoglobin levels to prevent
anemia-related morbidity and more accurate information on the effect of
iron-mediated organ damage on the outcome of patients with MDS.